Abstract
Muscular dystrophies are heritable and heterogeneous neuromuscular disorders characterized by the primary wasting of skeletal muscle, usually caused by mutations in the proteins forming the link between the cytoskeleton and the basal lamina. As a result of mutations in the dystrophin gene, Duchenne muscular dystrophy patients suffer from progressive muscle atrophy and an exhaustion of muscular regenerative capacity. No efficient therapies are available. The evidence that adult stem cells were capable of participating in the regeneration of more than their resident organ led to the development of potential stem cell treatments for degenerative disorder. In the present review, we describe the different types of myogenic stem cells and their possible use for the progression of cell therapy in Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy and unfortunately no effective therapy is available at present. As stem cells received much attention for their potential use in cell-based therapies for human diseases, herein we described multiple types of resident and circulating myogenic stem cells, their characterization and their possible use to treat muscular dystrophies.
| Original language | English |
|---|---|
| Pages (from-to) | 4251-4262 |
| Number of pages | 12 |
| Journal | FEBS Journal |
| Volume | 280 |
| Issue number | 17 |
| DOIs | |
| Publication status | Published - Sep 2013 |
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Keywords
- Duchenne muscular dystrophy
- stem cells
- therapy
ASJC Scopus subject areas
- Biochemistry
- Cell Biology
- Molecular Biology
Cite this
Perspectives of stem cell therapy in Duchenne muscular dystrophy. / Meregalli, Mirella; Farini, Andrea; Belicchi, Marzia; Parolini, Daniele; Cassinelli, Letizia; Razini, Paola; Sitzia, Clementina; Torrente, Yvan.
In: FEBS Journal, Vol. 280, No. 17, 09.2013, p. 4251-4262.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Perspectives of stem cell therapy in Duchenne muscular dystrophy
AU - Meregalli, Mirella
AU - Farini, Andrea
AU - Belicchi, Marzia
AU - Parolini, Daniele
AU - Cassinelli, Letizia
AU - Razini, Paola
AU - Sitzia, Clementina
AU - Torrente, Yvan
PY - 2013/9
Y1 - 2013/9
N2 - Muscular dystrophies are heritable and heterogeneous neuromuscular disorders characterized by the primary wasting of skeletal muscle, usually caused by mutations in the proteins forming the link between the cytoskeleton and the basal lamina. As a result of mutations in the dystrophin gene, Duchenne muscular dystrophy patients suffer from progressive muscle atrophy and an exhaustion of muscular regenerative capacity. No efficient therapies are available. The evidence that adult stem cells were capable of participating in the regeneration of more than their resident organ led to the development of potential stem cell treatments for degenerative disorder. In the present review, we describe the different types of myogenic stem cells and their possible use for the progression of cell therapy in Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy and unfortunately no effective therapy is available at present. As stem cells received much attention for their potential use in cell-based therapies for human diseases, herein we described multiple types of resident and circulating myogenic stem cells, their characterization and their possible use to treat muscular dystrophies.
AB - Muscular dystrophies are heritable and heterogeneous neuromuscular disorders characterized by the primary wasting of skeletal muscle, usually caused by mutations in the proteins forming the link between the cytoskeleton and the basal lamina. As a result of mutations in the dystrophin gene, Duchenne muscular dystrophy patients suffer from progressive muscle atrophy and an exhaustion of muscular regenerative capacity. No efficient therapies are available. The evidence that adult stem cells were capable of participating in the regeneration of more than their resident organ led to the development of potential stem cell treatments for degenerative disorder. In the present review, we describe the different types of myogenic stem cells and their possible use for the progression of cell therapy in Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy and unfortunately no effective therapy is available at present. As stem cells received much attention for their potential use in cell-based therapies for human diseases, herein we described multiple types of resident and circulating myogenic stem cells, their characterization and their possible use to treat muscular dystrophies.
KW - Duchenne muscular dystrophy
KW - stem cells
KW - therapy
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UR - http://www.scopus.com/inward/citedby.url?scp=84882725695&partnerID=8YFLogxK
U2 - 10.1111/febs.12083
DO - 10.1111/febs.12083
M3 - Article
C2 - 23206279
AN - SCOPUS:84882725695
VL - 280
SP - 4251
EP - 4262
JO - FEBS Journal
JF - FEBS Journal
SN - 1742-464X
IS - 17
ER -